Heidi Ledford in Nature:
When researcher Arkasubhra Ghosh finally met Uditi Saraf, he hoped that there was still a chance to save her. Ghosh and his collaborators were racing to design a one-off treatment that would edit the DNA in the 20-year-old woman’s brain cells and get them to stop producing toxic proteins. It was an approach that had never been tried before, with a long list of reasons for why it might not work. But the team was making swift progress. The researchers were maybe six months away from being ready to give Uditi the therapy, Ghosh told her parents over breakfast at their home outside New Delhi last June. Even so, Uditi’s mother was not satisfied. Work faster, she urged him.
Then, Uditi was carried to the breakfast table, and Ghosh understood her urgency. Once a gregarious and energetic child and teenager, with a quick laugh and a mischievous streak, Uditi was now unable to walk or feed herself. She had become nearly blind and deaf. Her family tried to talk to her: “These are the people who are making a therapy for you,” they said loudly. Shaken, Ghosh returned to his gene-therapy laboratory at Narayana Nethralaya Eye Hospital in Bengaluru, India, and got to work. “If you need to put up tents in the lab, then we can do so,” he told his students. “I’m not going to sleep.”
Four months later, Uditi was gone.
More here.